EU/3/08/528 - orphan designation for treatment chronic lymphocytic leukaemia

Lumiliximab

Overview

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in April 2010 on request of the sponsor.

On 4 February 2008, orphan designation EU/3/08/528 was granted by the European Commission to Biogen Idec Limited, United Kingdom, for lumiliximab for the treatment of chronic lymphocytic leukaemia.

What is chronic lymphocytic leukaemia?

Chronic lymphocytic leukaemia is a disease in which cancer cells are found in the blood and the bone marrow. The bone marrow is the spongy tissue inside the large bones in the body. Normally, the bone marrow makes cells, called “blasts”, which mature into several different types of blood cells with specific functions in the body. These include red cells, white cells and platelets. Red blood cells carry oxygen and other materials to all tissues of the body. White blood cells fight infection. Platelets support blood clotting. When leukaemia develops, the bone marrow produces large numbers of abnormal blood cells. Over a period of time these abnormal cells replace the normal white cells, red cells and platelets in the bone marrow, which reduces the number of normal cells in the blood and leads to anaemia, coagulation problems (bruising, haemorrhages) and repeated infections. There are several types of leukaemias. Chronic lymphocytic leukaemia is a cancer of a type of white blood cells called lymphocytes. The lymphocytes multiply and live too long, so there are too many of them circulating in the blood. These leukaemic lymphocytes look normal, but they are not fully developed and do not work properly. Chronic lymphocytic leukaemia is the most common type of leukaemia; it mainly affects older people, being rare in people under the age of 40 years. Chronic lymphocytic leukaemia is chronically debilitating and life-threatening, due to the severe prognosis and the poor long-term survival for high-risk patients.

What is the estimated number of patients affected by the condition?

At the time of designation, chronic lymphocytic leukaemia affected approximately 3.5 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 174,000 people, and is below the threshold for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).

* Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed based on data from the European Union (EU 27), Norway, Iceland and Lichtenstein. This represents a population of 498,000,000 (Eurostat 2006).

What treatments are available?

Treatment for leukaemia is complex, and depends on a number of factors including the type of leukaemia, the extent of the disease and whether the leukaemia has been treated before. It also depends on the age, symptoms, and the general health of the patient. Some people with chronic lymphocytic leukaemia never have treatment, if their illness is not causing any symptoms and is progressing slowly. Treatment is often started only if and when the symptoms become troublesome. Current main treatment of chronic lymphocytic leukaemia is chemotherapy (using drugs to kill cancer cells). Several products were authorised for the condition in the Community at the time of submission of the application for orphan drug designation.

Lumiliximab may be of potential significant benefit for the treatment of chronic lymphocytic leukaemia, because it might act differently from other medicinal products and may help kill cancer cells. This assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

How is this medicine expected to work?

Antibodies are proteins in the body that are able to recognise and bind to specific elements, the so called antigens. Antigens can be present on the surface of human cells (normal or cancer cells) or of microorganisms such as bacteria. Cancer cells typically have specific antigens in their surface; on the surface of the leukaemic cells, an antigen called CD23 is displayed. Lumiliximab is an antibody produced in a very specific way that results on antibodies with the same structure; these are called monoclonal antibodies. Lumiliximab has the ability to specifically recognise CD23 and therefore is able to bind to it. The union of lumiliximab and CD23 is ultimately thought to lead to a reaction in the cell that destroys the leukaemic cells bearing the CD23 molecule.

What is the stage of development of this medicine?

At the time of submission of the application for orphan designation, clinical trials in patients with chronic lymphocytic leukaemia were ongoing.

Lumiliximab was not authorised anywhere worldwide for chronic lymphocytic leukaemia or designated as orphan medicinal product elsewhere for this condition, at the time of submission.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 10 January 2008 recommending the granting of this designation.

Opinions on orphan medicinal products designations are based on the following cumulative criteria:

• the seriousness of the condition,
• the existence of alternative methods of diagnosis, prevention or treatment and
• either the rarity of the condition (affecting not more than 5 in 10,000 people in the European Union) or insufficient returns on investment.

Designated orphan medicinal products are products that are still under investigation and are considered for orphan designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.

EU/3/08/528: Public summary of positive opinion for orphan designation of lumiliximab for the treatment of chronic lymphocytic leukaemia

Adopted Reference Number: EMEA/COMP/36761/2008 Rev. 1

Key facts

• Active substance:
  Lumiliximab
• Intended use:
  Treatment chronic lymphocytic leukaemia
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/08/528
• Date of designation:
  03/02/2008
• Sponsor:

  Biogen Idec Limited; Thames House; Foundation Park; Maidenhead; Berkshire, SL6 3UD; United Kingdom; Telephone: + 44 1628 50 10 00; Telefax: + 44 1628 50 10 10; E-mail: [e-mail]